intra-arterial melphalan in treating younger patients with unilateral retinoblastoma

Conditions: Unilateral Retinoblastoma

Summary: this pilot clinical trial studies whether unilateral group d retinoblastoma, or retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can be treated with a new technique for delivering chemotherapy directly into the blood vessel that supplies the affected eye. this new technique is called intra-arterial injection. giving melphalan via intra-arterial injection may make it less likely that children will need surgery to remove the eye and may reduce the amount of treatment side effects.

primary objectives: i. to study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group d retinoblastoma, who would otherwise be considered for enucleation. secondary objectives: i. to estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group d disease. ii. to evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy. iii. to evaluate vision outcomes in children treated with intra-arterial chemotherapy. iv. to monitor the rate of the development of metastatic disease while on protocol therapy. tertiary objectives: i. to evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression. outline: patients receive melphalan intra-arterially (ia) on day 1. treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. after completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.

Eligibility criteria:
inclusion criteria: * newly diagnosed patients with unilateral group d retinoblastoma * magnetic resonance imaging (mri) (or computed tomography \[ct\] if mri is not available) of the brain must be performed within 14 days prior to study entry * diagnostic examination under anesthesia (eua) must be performed within 14 days prior to study entry * rapid central review confirmation of group d disease based on retcam images from diagnostic eua must be obtained before starting treatment * patients must have a performance status corresponding to eastern cooperative oncology group (ecog) scores of 0, 1 or 2 * patients must have a life expectancy of \>= 8 weeks * patients must have adequate renal function, defined as: * creatinine clearance or radioisotope glomerular filtration rate (gfr) \>= 70 ml/min/1.73 m\^2 or * a serum creatinine based on age/gender as follows: * 1 month to \< 6 months: 0.4 mg/dl * 6 months to \< 1 year: 0.5 mg/dl * 1 to \< 2 years: 0.6 mg/dl * 2 to \< 6 years: 0.8 mg/dl * 6 to \< 10 years: 1 mg/dl * 10 to \< 13 years: 1.2 mg/dl * 13 to \< 16 years: 1.5 mg/dl (male); 1.4 mg/dl (female) * \>= 16 years: 1.7 mg/dl (male); 1.4 mg/dl (female) * total bilirubin =\< 1.5 x upper limit of normal (uln) for age * serum glutamate pyruvate transaminase (sgpt) (alanine aminotransferase \[alt\]) \< 2.5 x upper limit of normal (uln) for age exclusion criteria: * patients with bilateral disease * unilateral retinoblastoma with group a, b, c, or e eyes * prior chemotherapy or radiation therapy for this disease (laser and cryotherapy are allowed and are not considered exclusion criteria) * clinical or neuroimaging evidence of extraocular disease or orbital optic nerve involvement